Unrelated Cord Blood Transplantation for Acute Leukemia Diagnosed in the First Year of Life: Outcomes and Risk Factor Analysis

Abstract

Infant acute leukemia still has a poor prognosis, and allogeneic hematopoietic stem cell transplantation is indicated in selected patients. Umbilical cord blood (UCB) is an attractive cell source for this population because of the low risk of chronic graft-versus-host disease (GVHD), the strong graft-versus-leukemia effect, and prompt donor availability. This retrospective, registry-based study reported UCB transplantation (UCBT) outcomes in 252 children with acute lymphoblastic leukemia (ALL; n = 157) or acute myelogenous leukemia (AML; n = 95) diagnosed before 1 year of age who received a single-unit UCBT after myeloablative conditioning between 1996 and 2012 in European Society for Blood and Marrow Transplantation centers. Median age at UCBT was 1.1 years, and median follow-up was 42 months. Most patients (57%) received a graft with 1 HLA disparity and were transplanted in first complete remission (CR; 55%). Cumulative incidence function (CIF) of day 100 acute GVHD (grades II to IV) was 40% ± 3% and of 4-year chronic GVHD was 13% ± 2%. CIF of 1-year transplant-related mortality was 23% ± 3% and of 4-year relapse was 27% ± 3%. Leukemia-free-survival (LFS) at 4 years was 50% ± 3%; it was 40% and 66% for those transplanted for ALL and AML, respectively (P = .001). LFS was better for patients transplanted in first CR, regardless of diagnosis. In multivariate model, diagnosis of ALL (P = .001), advanced disease status at UCBT (<.001), age at diagnosis younger than 3 months (P = .012), and date of transplant before 2004 were independently associated with worse LFS. UCBT is a suitable option for patients diagnosed with infant acute leukemia who achieve CR. In this cohort, patients with AML had better survival than those with ALL.

Hematopoietic cell transplantation in pediatric patients with acute leukemias or myelodysplastic syndrome using unrelated adult or umbilical cord blood donors in Brazil

“…Apart from this, very young children (<2‐3 year‐o) have intrinsically more aggressive leukemia, inadequate drug metabolism and besides that, usually do not receive TBI, which may have compromised the results in our ALL cohort, hampering UCBT results. This hypothesis is support by Ruggeri et al 17 who reported better survival for UCB younger children recipients with AML compared with those with ALL. In our study, however, relapse rates were similar among donor groups.…”

Impact of Consolidative Unrelated Cord Blood Transplantation on Clinical Outcomes of Patients With Relapsed/Refractory Acute B Lymphoblastic Leukemia Entering Remission Following CD19 Chimeric Antigen Receptor T Cells

“…Stem cells can be obtained from BM, PB, or UCB. UCB offers benefits such as rapid acquisition, less constricted HLA matching, and lower rates of GVHD ( 36 – 38 ). A comparative analysis of UCB and BM in children with acute leukemia demonstrated that 4-6/6 HLA-matched UCB provided a similar probability of LFS to matched BM ( 39 ).…”

Akraba Dışı Kordon Kanı Kaynaklı Kök Hücre Transplantasyonu Retrospektif Analizi

“…Başka bir çalışmada çoğu (%57'si) 1 HLA uyumsuzluğu olan greftten nakil olmuş 252 akut lösemi tanılı çocuk hastada %55 oranında tam remisyon sağlandığı bildirilmiştir. Söz konusu çalışmada bu hastaların %21'i ≥2'inde HLA uyumsuzluğu ve %22'sinde identik HLA uyumu mevcut olduğu, nötrofil engrafman oluşma yüzdesinin 0-1 HLA uyumsuz olanlarda %91; 2 HLA uyumsuzluğu olanlarda %88 olduğu bildirilmiştir (19). Buna benzer başka bir çalışmada, 504 akut lösemi tanılı erişkin ve genç erişkin vaka HLA uyumu açısından değerlendirildiğinde 0-1 HLA uyumsuzluğu olan hasta oranı %38,1; 2 veya 2' den fazla HLA uyumsuzluğu olan hasta oranı ise %61,9 olarak bildirilmiştir (20).…”